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Synthetic oil triheptanoin improves Rett syndrome, longevity

When young mice with the rodent equivalent of a rare autism spectrum disorder (ASD), called Rett syndrome, were fed a diet supplemented with the synthetic oil triheptanoin, they lived longer than mice...

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UCSD researchers implicate new gene in idiopathic or non-syndromic autism

With the help of mouse models, induced pluripotent stem cells (iPSCs) and the "tooth fairy," researchers at the University of California, San Diego School of Medicine have implicated a new gene in...

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Scientists receive NARSAD Distinguished Investigator Grants for mental health...

The Brain & Behavior Research Foundation today announced the award of NARSAD Distinguished Investigator Grants valued at $1.5 million to 15 scientists, who are full professors or the equivalent,...

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New nationwide effort seeks to find novel approaches to treat ASD,...

Some of the genetic diseases that can cause autism spectrum disorder (ASD) and intellectual disability (ID) are so rare that even physicians who specialize in treating them can't be certain they have...

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Newron, Zambon announce re-submission of safinamide NDA to FDA

Newron Pharmaceuticals S.p.A., a research and development company focused on novel CNS and pain therapies, and its commercial and development partner Zambon S.p.A., an international pharmaceutical...

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Findings may lay foundation for treatment of neurodegenerative diseases

This story starts in 1955, upon the death of Albert Einstein, when the pathologist charged with performing the famous scientist's autopsy stole his brain.

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UVA research identifies new role of immune system in Rett syndrome

The immune system is designed to protect us from disease. But what if it was malfunctioning? Would it make a disease worse? That appears to be the case with Rett syndrome, a neurodevelopmental...

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Immune system plays surprising role in the progression of Rett syndrome

New research by investigators at the University of Massachusetts Medical School suggests the immune system plays an unsuspected and surprising role in the progression of Rett syndrome, a severe...

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New 2015 Rettsyndrome.org awards announced

Rettsyndrome.org announces today that the Board of Trustees has awarded $1M to launch the Read-Through Program, further translational research in the area of Neuro-Habilitation, and fund clinical...

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Scientists and families to gather in The Woodlands, Texas to build up...

It takes a committed community to develop effective treatments for a new disease. With that hope, scientists and families will come together in a Houston suburb July 30- Aug. 2 to build up the...

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Study finds mechanism that identifies cause of intellectual disabilities in...

The term intellectual disability covers a large number of clinical entities, some with known cause and others of uncertain origin. For example Down syndrome is due to an extra copy of chromosome 21 and...

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RSAM, Rettsyndrome.org renew commitment to jointly defeat Rett syndrome

The Rett Syndrome Association of Massachusetts and Rettsyndrome.org announce today their renewed commitment to work together to defeat Rett syndrome. A generous $85,000 donation from RSAM will support...

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Researchers use stem cell-derived 'mini-brains' to identify drug candidate...

Using "mini-brains" built with induced pluripotent stem cells derived from patients with a rare, but devastating, neurological disorder, researchers at University of California, San Diego School of...

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New genetic discovery could lead to better treatment for X-linked diseases

Think back to middle school biology class, when you learned that boys have an X and a Y chromosome inside each cell, and girls have two X's.

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Study provides biochemical links between genes linked to autism and the...

A study led by the University of Utah School of Medicine provides new insights into how the subtle changes within cells, caused by disruptions in a gene called Kirrel3, could underlie some types of...

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Research reveals MECP2 Duplication Syndrome can be reversed

The methyl CpG binding protein 2 gene (MECP2) produces a protein of the same name, the level of which is critical for normal brain function. Mutations leading to protein under-expression cause Rett...

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Research could lead to new treatment for Rett Syndrome, other forms of autism

Penn State scientists have discovered a novel drug target and have rescued functional deficits in human nerve cells derived from patients with Rett Syndrome, a severe form of autism-spectrum disorder.

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LouLou Foundation, Penn Med set up Program of Excellence to develop...

The London-based LouLou Foundation and the Orphan Disease Center of the Perelman School of Medicine at the University of Pennsylvania have established a Program of Excellence to develop effective...

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DNA sequence features predict genome-wide binding pattern of key protein...

Researchers from the University of Illinois at Urbana-Champaign and the University of California-Davis are combining in vivo experimentation with computation for highly accurate prediction of the...

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Drexel study provides new insight into future treatment of breathing disorders

Bringing a steady supply of fresh air to the lungs can seem like a simple task, but breathing is a careful orchestration of brain and body.

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